455 Non-viral gene therapy for recessive dystrophic epidermolysis bullosa: Hyper branched aminated polyesters mediated minicircle DNA delivery

Abstract

Recessive dystrophic epidermolysis bullosa (RDEB) is a rare autosomal inherited skin blistering disorder caused by mutations in the type VII collagen gene (COL7A1). The large size of COL7A1 is a big delivery challenge for both viral and non-viral vectors and seriously hindered the development of therapeutic gene replacement therapy for RDEB. Our group has developed a non-viral minicircle DNA mediated gene replacement strategy for RDEB, which achieved high transfection efficiency while safety is improved by avoiding the usage of viruses and the removal of bacterial backbone from the plasmid DNA.